룩소리티닙 백반증 치료제 FDA 승인검토 연기

 

FDA에서 인싸이트사에 백반증 치료제로서의 검토 연기 명령

Published: Mar 14, 2022 By Mark Terry

Courtesy of Eyesonmilan/Shutterstock

 

 

월요일(2022. 3. 13) 인싸이트사는 FDA에서 옵젤루라(Opzelura: 룩소니티닙 크림)를 백반증 치료용으로 추가 신약신청한 것에 대하여 검토를 연기했다고 보고했다. FDA에서는 인싸이트사에서 제출한 3상 시험에 대하여 더 많은 데이터가 필요하고 이에 따라 검토에 추가시간이 필요하다고 하였다. 새로운 PDUFA 기한은 2022년 7월 18일이다.

 

** Prescription Drug User Fee Act (PDUFA)는 전문의약품 허가신청자 비용부담법으로, FDA에서 신약 허가 검토를 위해 지불하는 수수료를 명시하고 있음

 

옵젤루라는 인싸이트사의 선택적 JAK1/JAK2 억제제 룩소리티닙의 제형으로 12세 이상 경증~중증도의 아토피 피부염 치료제로 미국에서 승인된 유일한 국소 JAK 억제제이다.

또한 인싸이트사는 비분절성 백반증이 있는 12세 이상의 청소년 및 성인 600명 이상을 대상으로 연구하고 있다.

 

2021년 10월 2일, 인싸이트는 백반증 환자들에게 15% 룩소리티닙크림을 하루 2회 사용하는 치료를 24주간 실시한 결과 1차 및 2차 평가에서 위약보다 더 큰 치료결과가 나타났다고 발표했다.

 

24주차에 룩소리티닙 그룹 환자의 29.9%가 연구의 1차 평가변수인 안면 백반증 영역 점수 지수(F-VAS175)에서 기준선보다 75% 이상 개선되었고, 그룹의 51% 이상은 F-VASI50에서 기준선보다 50% 이상 개선되었으며, F-VASI90에서는 15% 이상이 주요 2차 평가변수인 90% 이상 개선되었다.

전반적인 안전성 면에서는 이전 연구와 마찬가지로 특별한 이상반응이 없었다.

 

당시 Tufts Medical Center의 Dermatology Department 연구 및 교육 부의장인 David Rosmarin 박사는 “중추 시험에서 임상적으로 의미 있는 색소 침착 개선과 이것이 내 환자에게 가져올 수 있는 잠재적 이점에 의해 입증된 바와 같이 룩소리티닙 크림의 잠재력에 대해 매우 고무된다.”라고 밝혔다.

 

 

<체크포인트>

1. 룩소리티닙 제형의 연고가 원래는 아토피 치료용으로 개발된 제품이었네요.

2. 백반증 환자를 대상으로 한 3상 시험 관련 데이터가 더 필요하다고 검토가 연기된 것이구요

3. 24주간(약 6개월) 하루 2회 사용한 결과 얼굴백반증의 75% 이상 회복된 환자가 29.9%라네요.

 

 

 

출처 : https://www.biospace.com/article/fda-orders-incyte-to-wait-for-review-of-vitiligo-treatment-/

 

 

 

<원문>

FDA Orders Incyte to Wait for Review of Vitiligo Treatment

Published: Mar 14, 2022 By Mark Terry

 

Courtesy of Eyesonmilan/Shutterstock

 

On Monday, Incyte Corporation reported that the U.S. Food and Drug Administration (FDA) had pushed back the review of the company’s supplemental New Drug Application (sNDA) for Opzelura (ruxolitinib cream) for vitiligo. The agency required additional time to review more data from the ongoing Phase III trial that the company submitted upon request. The new PDUFA date is July 18, 2022.

 

Opzelura is a formulation of Incyte’s selective JAK1/JAK2 inhibitor ruxolitinib. It’s the only topical JAK inhibitor approved in the U.S. for topical short-term and non-continuous chronic treatment of mild to moderate atopic dermatitis (AD) in patients 12 years of age and older who are non-immunocompromised and whose disease isn’t adequately controlled with other topical therapies.

 

The ongoing Phase III TRuE-V program, which includes TRuE-V1 and -V2, is studying the drug compared to vehicle (placebo) in more than 600 adolescents and adults aged 12 and older with non-segmental vitiligo. Vitiligo is a dermatological condition where the skin loses its melanocytes, or pigmented cells. This causes discolored patches of skin.

 

“We are confident in the data from the TRuE-V clinical trial program which supports our sNDA submission for ruxolitinib cream in vitiligo, and we look forward to bringing this innovative topical treatment to patients with vitiligo in the United States for whom there are no approved therapies that address repigmentation,” said Dr. Steven Stein, Incyte’s chief medical officer.

 

In September 2021, the FDA approved Opzelura cream for atopic dermatitis. At that time, it had previously had an earlier PDUFA date as well, but the FDA had requested more information. The agency then declared a major amendment and pushed back the target action date by three months.

 

Earlier that year, Incyte presented 52-week safety and efficacy data from the two Phase III TRuE-AD trials at the Revolutionizing Atopic Dermatitis (RAD) virtual symposium. That date was used for the atopic dermatitis approval. The studies met both the primary and key secondary endpoints, including patient-reported outcomes.

 

On Oct. 2, 2021, Incyte announced full results from the pivotal Phase III TRuE-V study of Opzelura in adolescents and adults aged 12 years and up with nonsegmental vitiligo. The Week 24 analysis demonstrating treatment with 15% ruxolitinib cream twice a day showed greater improvement than vehicle for all primary and key secondary endpoints in both the TRuE-V1 and –V2 studies.

 

At Week 24, 29.9% of patients in the ruxolitinib group achieved 75% or greater improvement from baseline in the facial Vitiligo Area Scoring Index (F-VAS175), which was the study’s primary endpoint. More than 51% of the ruxolitinib group had 50% or greater improvement from baseline in F-VASI50 and more than 15% hit 90% or higher in F-VASI90, key secondary endpoints.

 

The overall safety profile was consistent with previous studies, with no clinically significant application site reactions.

 

At the time, Vice-Chair of Research and Education at the Dermatology Department at Tufts Medical Center Dr. David Rosmarin stated, “The skin depigmentation associated with vitiligo can have a profound impact on a person’s life and patients are in need of additional treatment options. I am extremely encouraged about the potential of ruxolitinib cream, as evidenced by the clinically meaningful improvements in repigmentation shown in the pivotal trials, and the potential benefits it may bring for my patients.”

 

The company’s non-topical formulation of ruxolitinib is Jakafi, which is indicated for polycythemia vera, myelofibrosis, and acute and chronic graft-versus-host disease. In the fourth quarter of 2021, Jakafi brought in $592 million, for a total of $2.135 billion in yearly sales.

 

Opzelura wasn’t launched until late in 2021, but at the company’s year-end report, company chief executive officer Herve Hoppenot said, “Our launch of Opzelura has been very successful thus far. Nearly 19,000 patients were treated in the fourth quarter and feedback from both dermatologists and patients has been very positive. Importantly, we have also made significant progress towards ensuring optimal patient access to Opzelura.”